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PE20151774A1 - CONSTRUCTIONS OF MITOCHONDRIAL PROTEINS AND THEIR USES - Google Patents

CONSTRUCTIONS OF MITOCHONDRIAL PROTEINS AND THEIR USES

Info

Publication number
PE20151774A1
PE20151774A1 PE2015002144A PE2015002144A PE20151774A1 PE 20151774 A1 PE20151774 A1 PE 20151774A1 PE 2015002144 A PE2015002144 A PE 2015002144A PE 2015002144 A PE2015002144 A PE 2015002144A PE 20151774 A1 PE20151774 A1 PE 20151774A1
Authority
PE
Peru
Prior art keywords
constructions
disclosed
mitochondrial proteins
mitochondrial
mitochondria
Prior art date
Application number
PE2015002144A
Other languages
Spanish (es)
Inventor
Hagar Greif
Haya Lorberboum-Galski
Original Assignee
Bio Blast Pharma Ltd
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Bio Blast Pharma Ltd filed Critical Bio Blast Pharma Ltd
Publication of PE20151774A1 publication Critical patent/PE20151774A1/en

Links

Classifications

    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C07K14/46—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • C07K14/47—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61K—PREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K38/00—Medicinal preparations containing peptides
    • A61K38/16—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/02—Drugs for disorders of the nervous system for peripheral neuropathies
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/02—Ophthalmic agents
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P27/00—Drugs for disorders of the senses
    • A61P27/16—Otologicals
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P3/00—Drugs for disorders of the metabolism
    • A61P3/08—Drugs for disorders of the metabolism for glucose homeostasis
    • A61P3/10—Drugs for disorders of the metabolism for glucose homeostasis for hyperglycaemia, e.g. antidiabetics
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P39/00—General protective or antinoxious agents
    • A61P39/06—Free radical scavengers or antioxidants
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
    • A61P9/00—Drugs for disorders of the cardiovascular system
    • A61P9/04—Inotropic agents, i.e. stimulants of cardiac contraction; Drugs for heart failure
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/005—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from viruses
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K14/00—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof
    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/0004—Oxidoreductases (1.)
    • C12N9/0091—Oxidoreductases (1.) oxidizing metal ions (1.16)
    • C—CHEMISTRY; METALLURGY
    • C12—BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
    • C12N—MICROORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICROORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
    • C12N9/00—Enzymes; Proenzymes; Compositions thereof; Processes for preparing, activating, inhibiting, separating or purifying enzymes
    • C12N9/10—Transferases (2.)
    • C12N9/1003—Transferases (2.) transferring one-carbon groups (2.1)
    • C12N9/1018—Carboxy- and carbamoyl transferases (2.1.3)
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/07—Fusion polypeptide containing a localisation/targetting motif containing a mitochondrial localisation signal
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/01—Fusion polypeptide containing a localisation/targetting motif
    • C07K2319/10—Fusion polypeptide containing a localisation/targetting motif containing a tag for extracellular membrane crossing, e.g. TAT or VP22
    • C—CHEMISTRY; METALLURGY
    • C07—ORGANIC CHEMISTRY
    • C07K—PEPTIDES
    • C07K2319/00—Fusion polypeptide
    • C07K2319/20—Fusion polypeptide containing a tag with affinity for a non-protein ligand
    • C07K2319/21—Fusion polypeptide containing a tag with affinity for a non-protein ligand containing a His-tag
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S530/00—Chemistry: natural resins or derivatives; peptides or proteins; lignins or reaction products thereof
    • Y10S530/82—Proteins from microorganisms
    • Y10S530/826—Viruses
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10—TECHNICAL SUBJECTS COVERED BY FORMER USPC
    • Y10S—TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y10S530/00—Chemistry: natural resins or derivatives; peptides or proteins; lignins or reaction products thereof
    • Y10S530/827—Proteins from mammals or birds

Landscapes

  • Health & Medical Sciences (AREA)
  • Chemical & Material Sciences (AREA)
  • Life Sciences & Earth Sciences (AREA)
  • Organic Chemistry (AREA)
  • General Health & Medical Sciences (AREA)
  • Medicinal Chemistry (AREA)
  • Bioinformatics & Cheminformatics (AREA)
  • Engineering & Computer Science (AREA)
  • Genetics & Genomics (AREA)
  • Zoology (AREA)
  • Biochemistry (AREA)
  • Molecular Biology (AREA)
  • Pharmacology & Pharmacy (AREA)
  • Public Health (AREA)
  • Veterinary Medicine (AREA)
  • Animal Behavior & Ethology (AREA)
  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • General Chemical & Material Sciences (AREA)
  • Chemical Kinetics & Catalysis (AREA)
  • Wood Science & Technology (AREA)
  • Gastroenterology & Hepatology (AREA)
  • Proteomics, Peptides & Aminoacids (AREA)
  • Biophysics (AREA)
  • Biomedical Technology (AREA)
  • Toxicology (AREA)
  • Biotechnology (AREA)
  • General Engineering & Computer Science (AREA)
  • Microbiology (AREA)
  • Diabetes (AREA)
  • Cardiology (AREA)
  • Neurosurgery (AREA)
  • Neurology (AREA)
  • Virology (AREA)
  • Immunology (AREA)
  • Epidemiology (AREA)
  • Marine Sciences & Fisheries (AREA)
  • Hematology (AREA)
  • Ophthalmology & Optometry (AREA)
  • Obesity (AREA)
  • Hospice & Palliative Care (AREA)

Abstract

Se divulgan construcciones de proteinas de fusion que comprenden una proteina mitocondrial funcional, que pueden ingresar a la mitocondria dentro de las celulas intactas. Ademas, se divulgan metodos de tratamiento de trastornos mitocondriales con las proteinas de fusion divulgadas y composiciones correspondientesFusion protein constructs comprising a functional mitochondrial protein are disclosed, which can enter the mitochondria within intact cells. Furthermore, methods of treating mitochondrial disorders with the disclosed fusion proteins and corresponding compositions are disclosed.

PE2015002144A 2013-04-15 2014-04-10 CONSTRUCTIONS OF MITOCHONDRIAL PROTEINS AND THEIR USES PE20151774A1 (en)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201361811934P 2013-04-15 2013-04-15
US201361869981P 2013-08-26 2013-08-26
US14/034,224 US8912147B2 (en) 2013-04-15 2013-09-23 Mitochondrial proteins constructs and uses thereof

Publications (1)

Publication Number Publication Date
PE20151774A1 true PE20151774A1 (en) 2015-12-20

Family

ID=51686951

Family Applications (1)

Application Number Title Priority Date Filing Date
PE2015002144A PE20151774A1 (en) 2013-04-15 2014-04-10 CONSTRUCTIONS OF MITOCHONDRIAL PROTEINS AND THEIR USES

Country Status (13)

Country Link
US (1) US8912147B2 (en)
EP (1) EP2986629B1 (en)
JP (1) JP2016520557A (en)
KR (1) KR20150143778A (en)
AU (1) AU2014255313A1 (en)
BR (1) BR112015026017A2 (en)
CA (1) CA2909276A1 (en)
EA (1) EA201501028A1 (en)
MD (1) MD20150109A2 (en)
MX (1) MX2015014424A (en)
PE (1) PE20151774A1 (en)
SG (1) SG11201508313TA (en)
WO (1) WO2014170896A2 (en)

Families Citing this family (12)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
KR20180132833A (en) 2016-04-12 2018-12-12 이섬 리서치 디벨러프먼트 컴파니 오브 더 히브루 유니버시티 오브 예루살렘 엘티디. Methylmalonyl coenzyme A mutase (MCM) fusion constructs for the treatment of disorders associated with MCM deficiency
GB201721833D0 (en) * 2017-12-22 2018-02-07 Cancer Research Tech Ltd Fusion proteins
US20220096520A1 (en) * 2018-12-06 2022-03-31 Arcturus Therapeutics, Inc. Modified proteins and associated methods of treatment
ES2960692T3 (en) * 2018-12-06 2024-03-06 Arcturus Therapeutics Inc Compositions and methods for the treatment of ornithine transcarbamylase deficiency
CN110003345A (en) * 2019-04-03 2019-07-12 天津师范大学 A kind of the TAT-EseD recombinant protein and purposes of anti-Edwardsiella tarda
EP3999102A1 (en) 2019-07-18 2022-05-25 Larimar Therapeutics, Inc. Use of frataxin for treating leigh syndrome, french canadian type
AU2020321901A1 (en) 2019-07-29 2022-02-24 The Trustees Of Indiana University Materials and methods for treating Friedreich's Ataxia
AU2020391489A1 (en) 2019-11-25 2022-06-09 Larimar Therapeutics, Inc. Methods for quantifying frataxin activity
EP4114470A4 (en) 2020-03-03 2024-04-17 Arcturus Therapeutics, Inc. COMPOSITIONS AND METHODS FOR TREATING ORNITHINE TRANSCARBAMYLASE DEFICIENCY
CN115843269A (en) * 2020-03-26 2023-03-24 拉利玛生物医药公司 Molecules for organelle-specific protein delivery
CA3176208A1 (en) 2020-04-30 2021-11-04 Joan David Bettoun Methods for treating myelin associated diseases and mitochondria associated diseases
CN116867506A (en) 2020-12-12 2023-10-10 拉利玛生物医药公司 Pharmaceutical compositions including Friedreich's ataxin fusion proteins and methods of use

Family Cites Families (2)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
WO2005042560A2 (en) * 2003-10-24 2005-05-12 Wake Forest University Health Sciences Non-viral delivery of compounds to mitochondria
ES2560097T3 (en) 2008-02-04 2016-02-17 Yissum Research Development Company Of The Hebrew University Of Jerusalem Ltd. Procedures and compositions for treating mitochondrial disorders

Also Published As

Publication number Publication date
US8912147B2 (en) 2014-12-16
MD20150109A2 (en) 2016-03-31
US20140308262A1 (en) 2014-10-16
KR20150143778A (en) 2015-12-23
EP2986629B1 (en) 2017-11-08
EP2986629A2 (en) 2016-02-24
WO2014170896A3 (en) 2014-12-31
SG11201508313TA (en) 2015-11-27
AU2014255313A1 (en) 2015-09-10
JP2016520557A (en) 2016-07-14
CA2909276A1 (en) 2014-10-23
EA201501028A1 (en) 2016-04-29
WO2014170896A2 (en) 2014-10-23
MX2015014424A (en) 2016-05-18
BR112015026017A2 (en) 2017-10-17

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